RU2016150050A3 - - Google Patents
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- Publication number
- RU2016150050A3 RU2016150050A3 RU2016150050A RU2016150050A RU2016150050A3 RU 2016150050 A3 RU2016150050 A3 RU 2016150050A3 RU 2016150050 A RU2016150050 A RU 2016150050A RU 2016150050 A RU2016150050 A RU 2016150050A RU 2016150050 A3 RU2016150050 A3 RU 2016150050A3
- Authority
- RU
- Russia
Links
Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0085—Brain, e.g. brain implants; Spinal cord
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
- C12N2310/141—MicroRNAs, miRNAs
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted vectors
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Psychology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201462000895P | 2014-05-20 | 2014-05-20 | |
| US62/000,895 | 2014-05-20 | ||
| PCT/US2015/031783 WO2015179525A1 (en) | 2014-05-20 | 2015-05-20 | Huntington's disease therapeutic compounds |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| RU2016150050A RU2016150050A (ru) | 2018-06-21 |
| RU2016150050A3 true RU2016150050A3 (ja) | 2019-02-05 |
| RU2711147C2 RU2711147C2 (ru) | 2020-01-15 |
Family
ID=54554698
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2016150050A RU2711147C2 (ru) | 2014-05-20 | 2015-05-20 | Терапевтические соединения для лечения болезни хантингтона |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US9523093B2 (ja) |
| EP (1) | EP3146051B8 (ja) |
| JP (1) | JP6663859B2 (ja) |
| CN (1) | CN107075514A (ja) |
| AU (1) | AU2015264263B2 (ja) |
| CA (1) | CA2949437C (ja) |
| ES (1) | ES2759317T3 (ja) |
| RU (1) | RU2711147C2 (ja) |
| WO (1) | WO2015179525A1 (ja) |
| ZA (1) | ZA201608108B (ja) |
Families Citing this family (52)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
| SG171914A1 (en) | 2008-12-02 | 2011-07-28 | Chiralgen Ltd | Method for the synthesis of phosphorus atom modified nucleic acids |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| AU2010270714B2 (en) | 2009-07-06 | 2015-08-13 | Wave Life Sciences Ltd. | Novel nucleic acid prodrugs and methods use thereof |
| DK2561073T3 (en) | 2010-04-23 | 2016-12-12 | Univ Massachusetts | Aav vectors targeted to central nervous system and methods of use thereof |
| WO2011133874A1 (en) | 2010-04-23 | 2011-10-27 | University Of Massachusetts | Multicistronic expression constructs |
| US10428019B2 (en) | 2010-09-24 | 2019-10-01 | Wave Life Sciences Ltd. | Chiral auxiliaries |
| WO2012145624A2 (en) | 2011-04-21 | 2012-10-26 | University Of Massachusetts | Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies |
| DK2734208T3 (en) | 2011-07-19 | 2017-06-19 | Wave Life Sciences Ltd | PROCEDURES FOR SYNTHESIS OF FUNCTIONALIZED NUCLEIC ACIDS |
| JP6246121B2 (ja) | 2012-07-13 | 2017-12-13 | 株式会社新日本科学 | キラル核酸アジュバント |
| SG11201500232UA (en) | 2012-07-13 | 2015-04-29 | Wave Life Sciences Pte Ltd | Chiral control |
| US9598458B2 (en) | 2012-07-13 | 2017-03-21 | Wave Life Sciences Japan, Inc. | Asymmetric auxiliary group |
| JPWO2015108047A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤 |
| EP3095460A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent |
| JPWO2015108048A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤 |
| US10160969B2 (en) | 2014-01-16 | 2018-12-25 | Wave Life Sciences Ltd. | Chiral design |
| WO2015143078A1 (en) | 2014-03-18 | 2015-09-24 | University Of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
| AU2015264263B2 (en) | 2014-05-20 | 2021-08-05 | University Of Iowa Research Foundation | Huntington's disease therapeutic compounds |
| WO2015187825A2 (en) | 2014-06-03 | 2015-12-10 | University Of Massachusetts | Compositions and methods for modulating dysferlin expression |
| EP3200830B1 (en) | 2014-10-03 | 2020-09-09 | University of Massachusetts | High efficiency library-identified aav vectors |
| WO2016054554A1 (en) | 2014-10-03 | 2016-04-07 | University Of Massachusetts | Heterologous targeting peptide grafted aavs |
| CA2964272A1 (en) | 2014-10-21 | 2016-04-28 | Guangping Gao | Recombinant aav variants and uses thereof |
| KR20230145206A (ko) | 2014-11-14 | 2023-10-17 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| BR112017010087A2 (pt) | 2014-11-14 | 2018-06-05 | Voyager Therapeutics, Inc. | composições e métodos para tratar esclerose lateral amiotrófica (ela) |
| US10435467B2 (en) | 2015-01-08 | 2019-10-08 | Biogen Ma Inc. | LINGO-1 antagonists and uses for treatment of demyelinating disorders |
| EP3256170B1 (en) | 2015-02-13 | 2020-09-23 | University of Massachusetts | Compositions and methods for transient delivery of nucleases |
| US11046955B2 (en) | 2015-04-24 | 2021-06-29 | University Of Massachusetts | Modified AAV constructs and uses thereof |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| EP3364996B1 (en) | 2015-10-22 | 2021-08-25 | University of Massachusetts | Prostate-targeting adeno-associated virus serotype vectors |
| US11253576B2 (en) | 2015-10-22 | 2022-02-22 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance in neurodegenerative disease |
| EP3411059A4 (en) | 2016-02-02 | 2019-10-16 | University Of Massachusetts | METHOD FOR INCREASING THE EFFECTIVENESS OF THE SYSTEMIC DELIVERY OF AVV GENES TO THE CENTRAL NERVOUS SYSTEM |
| WO2017139643A1 (en) | 2016-02-12 | 2017-08-17 | University Of Massachusetts | Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization |
| WO2017176929A1 (en) | 2016-04-05 | 2017-10-12 | University Of Massachusetts | Compositions and methods for selective inhibition of grainyhead-like protein expression |
| US11413356B2 (en) | 2016-04-15 | 2022-08-16 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance |
| MA45270A (fr) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| RU2764587C2 (ru) | 2016-05-18 | 2022-01-18 | Вояджер Терапьютикс, Инк. | Способы и композиции для лечения хореи гентингтона |
| MX2018014154A (es) | 2016-05-18 | 2019-05-06 | Voyager Therapeutics Inc | Polinucleotidos moduladores. |
| WO2017218852A1 (en) | 2016-06-15 | 2017-12-21 | University Of Massachusetts | Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells |
| US10457940B2 (en) * | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
| EP3526333A4 (en) | 2016-10-13 | 2020-07-29 | University of Massachusetts | AAV CAPSIDE DESIGNS |
| EP3619308A4 (en) | 2017-05-05 | 2021-01-27 | Voyager Therapeutics, Inc. | COMPOSITIONS AND METHODS OF TREATMENT FOR HUNTINGTON'S MORBUS |
| AU2018261790A1 (en) | 2017-05-05 | 2019-11-28 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
| JP7327803B2 (ja) | 2017-05-09 | 2023-08-16 | ユニバーシティ オブ マサチューセッツ | 筋萎縮性側索硬化症（ａｌｓ）を処置する方法 |
| AU2018338188A1 (en) | 2017-09-22 | 2020-04-02 | University Of Massachusetts | SOD1 dual expression vectors and uses thereof |
| AR113134A1 (es) * | 2017-09-22 | 2020-01-29 | Genzyme Corp | Arni variante |
| EP4124658A3 (en) | 2017-10-16 | 2023-04-19 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| US11679129B2 (en) | 2018-01-12 | 2023-06-20 | Curocell, Inc. | Enhanced immune cells using dual shRNA and composition including the same |
| JP7333079B2 (ja) * | 2018-03-19 | 2023-08-24 | 国立大学法人 東京医科歯科大学 | 毒性が軽減した核酸 |
| JP7432929B2 (ja) | 2018-05-31 | 2024-02-19 | コリア ユニバーシティ リサーチ アンド ビジネス ファウンデーション | マイクロｒｎａの非正規標的を抑制するｒｎａ干渉誘導核酸およびその用途 |
| JP2021529513A (ja) | 2018-07-02 | 2021-11-04 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | 筋萎縮性側索硬化症および脊髄に関連する障害の治療 |
| JP2021530548A (ja) | 2018-07-24 | 2021-11-11 | ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics, Inc. | 遺伝子治療製剤を生産するための系および方法 |
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| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US4962091A (en) | 1986-05-23 | 1990-10-09 | Syntex (U.S.A.) Inc. | Controlled release of macromolecular polypeptides |
| US5585362A (en) | 1989-08-22 | 1996-12-17 | The Regents Of The University Of Michigan | Adenovirus vectors for gene therapy |
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| CA2327208A1 (en) | 2000-11-30 | 2002-05-30 | The Government Of The United States Of America | Methods of increasing distribution of therapeutic agents |
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| US9272016B2 (en) * | 2012-04-20 | 2016-03-01 | University Of Iowa Research Foundation | Methods to enhance RNAi oligonucleotide delivery to respiratory epithelial cells |
| AU2013287261B2 (en) * | 2012-07-06 | 2019-03-07 | University Of Iowa Research Foundation | Modified adeno-associated virus vector compositions |
| AU2015264263B2 (en) | 2014-05-20 | 2021-08-05 | University Of Iowa Research Foundation | Huntington's disease therapeutic compounds |
-
2015
- 2015-05-20 AU AU2015264263A patent/AU2015264263B2/en active Active
- 2015-05-20 RU RU2016150050A patent/RU2711147C2/ru active
- 2015-05-20 WO PCT/US2015/031783 patent/WO2015179525A1/en active Application Filing
- 2015-05-20 JP JP2016568589A patent/JP6663859B2/ja active Active
- 2015-05-20 US US14/717,795 patent/US9523093B2/en active Active
- 2015-05-20 EP EP15795373.8A patent/EP3146051B8/en active Active
- 2015-05-20 ES ES15795373T patent/ES2759317T3/es active Active
- 2015-05-20 CA CA2949437A patent/CA2949437C/en active Active
- 2015-05-20 CN CN201580039020.2A patent/CN107075514A/zh active Pending
-
2016
- 2016-11-23 ZA ZA2016/08108A patent/ZA201608108B/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| EP3146051A1 (en) | 2017-03-29 |
| RU2711147C2 (ru) | 2020-01-15 |
| AU2015264263A1 (en) | 2016-12-08 |
| US9523093B2 (en) | 2016-12-20 |
| WO2015179525A1 (en) | 2015-11-26 |
| JP6663859B2 (ja) | 2020-03-13 |
| RU2016150050A (ru) | 2018-06-21 |
| EP3146051A4 (en) | 2017-12-20 |
| CA2949437A1 (en) | 2015-11-26 |
| CA2949437C (en) | 2023-08-15 |
| ZA201608108B (en) | 2022-05-25 |
| AU2015264263B2 (en) | 2021-08-05 |
| ES2759317T3 (es) | 2020-05-08 |
| JP2017522005A (ja) | 2017-08-10 |
| US20160060624A1 (en) | 2016-03-03 |
| EP3146051B1 (en) | 2019-09-04 |
| CN107075514A (zh) | 2017-08-18 |
| EP3146051B8 (en) | 2019-11-27 |